肌萎缩侧索硬化症治疗措施的临床证据评价

Evidence based evaluation of therapeutic measures for amyotrophic lateral sclerosis

目的评价不同治疗方案对肌萎缩侧索硬化症的作用及药物不良反应,以为循证制定最佳治疗方案。方法以肌萎缩侧索硬化症、利鲁唑、加巴喷丁、拉莫三嗪、神经营养因子、抗氧化和氧自由基清除药、基因治疗、神经干细胞治疗、治疗等词组作为检索词,分别检索MEDLINE、Cochrane图书馆、万方数据知识服务平台学术期刊库和中国知网中国期刊全文数据库,按照相应的纳入标准和排除标准,获取临床指南、系统评价、随机对照临床试验、临床对照试验、回顾性病例分析及病例观察研究的相关文献,并采用Jadad量表对文献质量进行评价,判断研究结论的真实性和可靠性。同时手工检索有关会议资料和中英文原著、教科书,并与该研究领域从事此方面研究的学者联系,以便对在线检索不到全文者直接Email联系以获取全文。结果经筛选纳入与肌萎缩侧索硬化症治疗相关的系统评价4篇、随机对照临床试验18篇、临床对照试验11篇、病例观察研究6篇,其中27篇为高质量文献(Jadad量表评价:11篇4分、13篇5分、3篇7分),12篇为低质量文献(评分3分)。对各种治疗方法或药物疗效及安全性进行评价显示:利鲁唑为目前唯一被美国食品与药品管理局批准应用于临床的药物,缺乏其他更有效的治疗药物;利鲁唑无效时可采用多药联合方案及对症治疗。结论借助循证医学评价方法可为肌萎缩侧索硬化症患者的治疗提供最佳临床证据。

Objective To evaluate the therapeutic efficacy and side effects of various treatments for amyotrophic lateral sclerosis （ALS） in order to formulate the best therapeutic regimen. Methods ALS, Riluzole, Gabapentin, Lamotrigine, neurotrophic factor, antioxidant and free radical scavenger gene therapy, neural stem cell treatment, treatment were appointed as retrieval words. MEDLINE, Cochrane Library, Wanfang Database for Scientific Journals in China and Chinese National Knowledge Infrastructure （CNKI） for Scientific Journals Database were used for retrieval. Related clinical guidelines, systematic reviews, randomised controlled trials, controlled clinical trials and case-observation studies were collected following the corresponding inclusion criteria and exclusion criteria and evaluated by Jadad Scale to judge the authenticity and reliability of the conclusion. Manual searching was also used. Results After screening, 39 related articals were selected as follow： 4 systematic reviews, 18 randomised controlled trials, 11 controlled clinical trials, and 6 case- observation studies. Twenty- seven articals were of high quality （according to Jadad Scale, 11 with score 4, 13 with score 5, and 3 with score 7）, while 12 were of low quality with score 3. According to the evaluation of therapeutic efficacy and side effects of various therapies, it is suggested that： 1） The Riluzole is the only drug approved by American FDA, lacking of more effective treatments. 2） When the patients condition is not relieved, medicine-combined therapies and symptomatic treatments should be used. Conclusion Evidence-based medicine can provide best clinical evidence on ALS treatment.